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Titlebook: Recent Advances In Retinal Degeneration; Robert E. Anderson,Matthew M. LaVail,Joe G. Hollyf Book 2008 The Editor(s) (if applicable) and Th

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楼主: 适婚女孩
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of the Retina in the 4 Years Following rAAV-mediated Gene Transfer in the RPE65 Null Mutation Dogvement following a single unilateral subretinal injection of a recombinant adeno-associated virus vector, serotype 2, rAAV2/2, cDNA for dog RPE65 with a cytomegalo virus (CMV) promoter (Narfström et al., 2003a,b, 2005; Ford et al., 2003). The purpose of the present investigation was to further asses
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Virus-mediated Gene Delivery to Neuronal Progenitorsredsson et al., 2006). Degenerations due to recessive mutations can be slowed or prevented by gene augmentation in which the wild-type form of the transgene of interest is packaged into a recombinant virus. Neurotrophic or neuroprotective factors can be delivered to tissues of interest by transducti
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Book 2008ng in the field. The topics in this volume explore the etiology, cellular mechanisms, epidemiology, generics, models and potential therapeutic measures for the blinding diseases of retinitis pigmentosa and age-related macular degeneration...Special focus is highlighted in the areas of Mechanisms of
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Virus-mediated Gene Delivery to Neuronal Progenitorson with an appropriate virus. And, in the case of dominant inherited degenerations, a recombinant virus carrying siRNA specific to the mutant transgene could be introduced to the cells of interest to result in decreased production of the dominant negative form of the protein.
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Retinal Degenerations: Planning for the Futuregeneration with a decrease in central vision after age 60. About 100,000 people have retinitis pigmentosa or a related disease with loss of side and night vision in adolescence; they often develop tunnel vision by age 40 and lose central vision by age 60.
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Gene Therapy for Mouse Models of ADRP RHO gene. Two strategies for . mRNA silencing have recently been proposed (Millington-Ward et al., 1997; Lewin and Hauswirth, 2001). They are based on the application of therapeutic molecules such as ribozymes (Rz) and small interfering RNA (siRNA) that inhibit . expression.
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Adeno-Associated Viral Vectors and the Retinaral strategies have been attempted to rescue retinal disease, including gene replacement (Acland et al., 2001), gene knockdown with both ribozymes (Gorbatyuk et al., 2007) and siRNA (Kiang et al., 2005), and therapeutic gene supplementation (Deng et al., 2005).
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Alexis-Pierre Bemelmans,Corinne Kostic,Maité Cachafeiro,Sylvain V. Crippa,Dana Wanner,Meriem Tekaya,
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